A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Status:
NOT_YET_RECRUITING
Trial end date:
2032-02-29
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212. It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA.
All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.